Duchenne UK is delighted to announce that seven leading pharmaceutical companies developing treatments for Duchenne Muscular Dystrophy, are collaborating with the charity on Project HERCULES, to develop a robust and relevant evidence base for payers to accelerate the time it takes for payers to make decisions about which medicines to fund. Duchenne UK is investing £200,000 in the initial stages of the project.

Project HERCULES aims to provide a core central evidence base to provide high quality material to enable an informed HTA process and enable smoother and speedier payer reviews for innovative treatments in Duchenne muscular dystrophy (DMD). The project will allow pharmaceutical companies to work together to build the evidence base for new therapies required by pricing watchdogs, such as the National Institute of Health and Care Excellence (NICE).

With a wave of treatments in, or about to enter clinical trials, and with some already gaining approval, the aim of HERCULES is to address the next challenge after medicines reach regulatory approval; how we get governments and health services to pay for these medicines.

It is widely recognised that the pace of innovation in treatments and medicines is outstripping the time it takes to approve them for use in the NHS. These delays are worsened for diseases as there is often limited evidence available to meet the requirements for access and pricing decisions.

In order to get NICE to recommend that the NHS can fund their medicines, companies are required to develop a vast amount of highly specialized and detailed work which includes data collection, economic modelling and quality of life measurements, all of which can be difficult, time consuming and expensive. The processes of bodies like NICE to assess the cost effectiveness of a medicine are well established and need to be followed. However, we hope that through the collaboration in HERCULES, we can help industry better engage with this process, present relevant evidence as required and therefore enable the decision making process, and potentially speed up the time it takes for industry to engage with bodies like NICE to make decisions

HERCULES comes from a desire to help develop industry to develop this disease level evidence in collaboration, providing benefits which vary from improved modelling (reducing uncertainty), a broader evidence base, reduction of generating evidence which is duplicative or inconsistent, improved engagement with patients, lower costs and management time, additionally being ethically superior by making better use of patient input.

Duchenne UK is investing £200,000 in the initial stages of the project,

which will help companies to work together to develop a SHARED model that will focus on developing common tools and practices, including:

“We are delighted to be able to launch HERCULES with the support of seven industry partners, all of whom are developing treatments for DMD. In order to persuade government bodies to pay for new medicines, companies often have to provide complex data analysis and burden of care studies which are costly and time-consuming. The aim of HERCULES is for companies to collaborate together on building such models, to save time, money and ensure that the collaboration speeds up access for patients.”

Josie Godfrey, ex-Director of NICE, who is running Hercules says:

“HTA agencies can struggle with decisions in the absence of a solid evidence base, be that data on the burden of the disease or the likely disease progression. Through its collaboration with patient organisations, industry, universities and others partners, Hercules will ensure all companies can produce the best possible evidence for HTA agencies – making decisions easier, fairer and hopefully enabling faster access to the best new treatments for DMD.”

“I’m thrilled that this initiative for collaboration across companies to support successful reimbursement is launching with Duchenne UK. It is a testament to the commitment of the companies to the Duchenne community to come together in this way and generate world-class disease level evidence for payers”

Project HERCULES coincides with the Accelerated Access Pathway initiative, which provides a fast-track route to getting ‘breakthrough’ treatments to patients up to four years earlier. This is part of the Government’s efforts to address the long-held problem that patients often get a raw deal when accessing the latest treatments.

Phase One of Project HERCULES has begun, and working with leading academic groups will focus on understanding and synthesizing the current evidence base, the cost and burden of DMD and lives lost to it, and current measures in DMD clinical trials. The project will also look at how quality of life for boys with DMD is measured and develop a new measure as the gold standard.

Following this, the Project will map the impact of DMD on patients, families and carers, and develop a disease level economic model to support pricing and reimbursement decisions. Phase Two will be announced in 2018.

Emily, Josie and Fleur discussing Project HERCULES in a panel discussion at the World Orphan Drug Congress in Barcelona, November 2017:

- ENDS -

Q & A on Hercules:

What is Project HERCULES?

Project HERCULES is the name given to a collaboration between Duchenne UK, and pharmaceutical companies who are developing medicines to treat Duchenne Muscular Dystrophy, DMD, to increase the chances of patients with DMD of accessing innovative treatments.

With a wave of treatments in, or about to enter clinical trials, and with some already gaining approval, the aim of HERCULES is to address the next challenge after medicines are approved: how we get governments to pay for these medicines. This is called reimbursement.

In order to persuade government bodies to pay for new medicines, companies often have to provide complex data analysis and burden of care studies which are costly and time-consuming. The aim of HERCULES is for companies to collaborate together on building such models, to save time, money and ensure that the collaboration speeds up access for patients.

What does HERCULES stand for?HERCULES stands for HEalth Research Collaboration United in Leading Evidence Synthesis.

Why do we need HERCULES?

We all share the hope that new medicines coming through will improve the lives of those living with DMD and their families. However, we have concerns about how difficult it has been to get countries to make decisions about whether or not to pay for these new medicines.

And we’re concerned that changes to these procedures in the UK could further prevent or delay those with DMD with access to effective new treatments.

Organisations like NICE, the National Institute of Clinical Excellence (NICE) in England, run what’s called Health Technology Assessments

(HTA) to determine whether new medicines offer value for money and should be paid for, known as reimbursement, on the NHS.

In order to get NICE to pay for their medicines, companies are required to develop a vast amount of highly specialized and detailed work which includes data collection, economic modelling and quality of life measurements, all of which can be difficult, time consuming and expensive. The processes are well established and need to be followed. However, we hope that through the collaboration in Hercules, we can help industry better engage with this process and actually speed up the time it takes for industry to engage with bodies like NICE to make decisions

What we propose with HERCULES, is to bring companies together to develop a SHARED model that will focus on developing common tools and practices, including: *Data analysis, including mapping clinical trial endpoints to clinical outcomes

We recognise that many patient organisations are active in this area and will be having conversations with HTA agencies and other agencies over the coming months. HERCULES is intended to complement and build on the work already underway within the DMD community. Project HERCULES aim is to provide both practical and tactical support to companies, and to also to all patient groups, so that interactions with HTA bodies are cohesive, persuasive and supported with a strong and relevant evidence base.

Who is running project HERCULES?

Duchenne UK is delighted that, Josie Godfrey, a former Associate Director at NICE, who set up the Highly Specialised Technologies Appraisals System, is running HERCULES. The Steering Group will be chaired by Fleur Chandler who works in Health Value Outcomes at GSK and who also sits on the Patient Advisory Board of Duchenne UK. Together they bring much valuable experience of HTA methods and processes.

Duchenne UK is extremely grateful to Anthony Hatswell, Professor Ron Akehurst and the Bresmed Foundation for their early support for the project.

We would also like to thank the legal team at CMS for their advice and support.

NOTES FOR EDITORS

What is Duchenne Muscular Dystrophy?

Duchenne Muscular Dystrophy is the most common fatal genetic disease diagnosed in childhood. Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. It almost exclusively affects boys. There is no treatment or cure. In the UK there are around 2,500 boys affected and around 300, 000 worldwide. It is classified as a rare disease.

Who are Duchenne UK?

Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy. The charity has been formed by the coming together of Joining Jack and Duchenne Children's Trust, the two biggest funders of research in the UK in the last three years. Its president is HRH The Duchess of Cornwall. Its patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell.

How to donate?

Duchenne UK is entirely reliant on donations to fund research for treatments and a cure to DMD. This can be done via:

Most read

Duchenne affects approximately 1 in every 3,500 boys that are born but only around 1 in every 50 million girls. It may be rare, but it does happen. We have been speaking to Feriel, a 26 year old woman living with Duchenne muscular dystrophy. She has written us a short blog about her experiences with Duchenne from diagnosis to now.

Our co-founders Alex Johnson and Emily Crossley met after their sons were diagnosed with Duchenne muscular dystrophy. They both set up charities, Alex with Joining Jack, Emily with the Duchenne Children’s Trust.

We are proud to share with you our first ever Impact Report, read about the very real impact we are having and the many things we have been working on over the past six years to end Duchenne.
Read more

We will always store your personal details securely. We’ll use them to provide the service that you have requested, and communicate with you in the way(s) that you have agreed to. Your data may also be used for analysis purposes, to help us provide the best service possible. For full details see our Privacy Policy or contact us on [email protected]